New Lab Models Accelerate Research for Pediatric Brain Tumors

Scientists at St. Jude Children's Research Hospital have developed new laboratory models for studying pediatric embryonal brain tumors. The models, which include patient-derived tumor organoids and tumor organoid xenografts, are designed to accelerate research into potential therapies. The findings were published in the journal Science Advances on April 12, 2026.

The Challenge: Hindered Treatment Development

Pediatric brain and central nervous system tumors are a leading cause of disease-related death in children in the United States.

According to the researchers, efforts to develop new treatments have been hindered by the limitations of existing laboratory models. Traditional models, such as patient-derived orthotopic xenografts, are noted for being expensive and time-intensive to establish, sometimes requiring months to develop.

The St. Jude team aimed to create models that would be more accessible and faster to use for functional assays and preclinical drug testing.

The Solution: Faster, More Accessible Models

The researchers developed two types of models:

• 3D tumor organoids grown in a laboratory setting.
• Tumor organoid xenografts, which are organoids implanted into immunodeficient mice.

The models were created using several types of pediatric embryonal brain tumors, including medulloblastoma, embryonal tumor with multilayer rosettes, and atypical teratoid rhabdoid tumors.

To validate the models, the team conducted molecular analyses, including DNA methylation profiling, bulk and single-cell RNA sequencing, and whole-genome sequencing. These analyses indicated that the models maintain the genetic, epigenetic, and cellular diversity of the original patient tumors from which they were derived.

In drug testing experiments, the response of the lab-grown organoids was similar to that of the corresponding tumor organoid xenografts.

Researcher Perspective and Model Sharing

"Some traditional patient-derived tumor models can take months to develop, which slows research progress and increases costs," stated corresponding author Martine Roussel, PhD.

Roussel said the newly developed organoid models provide a faster, more accessible way for researchers to study these tumors and test potential therapies.

Roussel also noted that not all research institutions have the resources to develop such complex models. To address this, the St. Jude team is making the organoid models available to other researchers upon request, with the aim of allowing more scientists to advance the study of pediatric brain tumors.

Study Details

The study's first author is Justin Williams of St. Jude Children's Research Hospital. Other authors include Dana Farmer, Qianqian Li, Jose Grenet, Sarah Robinson, Kimberly Mercer, Vanshita Goel, Laura Janke, Liusheng He, Paul Klimo, Jason Chiang, Giles Robinson, Brent Orr, and Jake Friske.

The research was supported by grants from the National Cancer Institute (CA021765 and CA096832), the St. Jude Graduate School of Biomedical Sciences, and the American Lebanese Syrian Associated Charities (ALSAC).